UNC grad with rare ALS strain can’t get drug she needs. She seeks social media’s help.

The first symptom appeared in August, when Lisa Stockman Mauriello noticed a hoarseness in her voice.

“People thought she had a bad night,” said her husband, Bob. “Maybe a little under the weather.”

Then came difficulty breathing, a scary escalation, until January, when the 51-year-old mother of three learned she had a rare and fast-moving form of ALS, or Lou Gehrig’s disease, that strikes the face first rather than the arms and legs.

“I cried all the way home from the hospital,” Lisa said Wednesday, as Bob translated her faint speech.

A drug and a hope to buy time

Last month, she learned about Tofersen, a promising drug in the last stages of testing that she hopes will buy more time.

But despite pleas from her doctor, the pharmaceutical giant Biogen will not provide the investigative drug before its FDA approval. After repeated requests, the family has gotten no explanation.

So Stockman Mauriello, an Apex High School and UNC-Chapel Hill graduate who often lectures at her alma mater, started a petition on change.org, describing her deep desire to buy a few more months.

Her oldest son, Scott, graduates from UNC in May. Her middle son, Luke, starts his Tar Heel years this fall. Her youngest son, Dean, will soon complete eighth grade — all milestones she hopes to witness.

“We don’t want this to be a hit piece on Biogen,” Bob Mauriello said from their home in New Jersey. “We’re thankful they’re doing the research. But in her case, were talking about months, and waiting until this summer or this fall or next year, it’s going to be too late.”

Amyotrophic lateral sclerosis, or ALS, is a neurological disease that attacks neurons controlling muscle movement. The Centers for Disease Control estimates roughly 15,000 Americans have ALS, for which there is no cure.

Stockman Mauriello, though, has been diagnosed with the bulbar form of ALS, which strikes at speech and swallowing and triggers far faster deterioration.

Four times a day, her husband feeds her through a tube. Speaking is difficult, and she often writes to communicate. A ventilator helps her sleep at night. Meanwhile, she can’t move her left arm, has lost much of the mobility in her right arm and walks with a limp, quickly becoming exhausted.

“There have definitely been joyful and appreciative moments,” said Bob Mauriello, also a UNC graduate. “But there are other moments when you think you’re not going to see our kids get married or see our first grandchild. A range of emotions, as you can imagine. We’re trying to enjoy the time we have together. No wallowing.”

Online petition

Tofersen is an artificial piece of DNA designed to slow the pace of ALS. Now in phase 3 of clinical trials, the last before application for FDA approval, it has showed promising signs, according to Massachusetts General Hospital.

The FDA provides guidelines for “compassionate use,” which allow patients to access investigative drugs when their lives are threatened and no other treatment exists.

But without support from Biogen, the family does not expect the FDA to sign off.

“My brother-in-law last night offered to drive up to Boston to the CEO of Biogen with a train of people,” Bob Mauriello said. “When you’re right there, it’s harder to say no.”

By Wednesday morning, the change.org petition had gathered more than 37,000 signatures, noting Stockman Mauriello’s personal plea to Biogen CEO Michael Vounatsos.

Hers is not the only plea for compassionate use now circulating online, and the company offered this response on Twitter on Monday: “Our hearts break for all families impacted by ALS, a devastating neurological condition. We evaluate each individual request for access to investigational therapies.”

On Wednesday, Bob Mauriello said the Biogen route may be blocked for them, and they are starting to look at other clinical trials that aren’t as far along.

Stockman Mauriello, meanwhile, is savoring a recent visit to the UNC campus, where the family set up a makeshift ballroom for mother-son dances.

One by one, she danced with all three, moving as best she could.

If she had her say, those minutes would last forever.

This story was originally published March 17, 2021 at 11:48 AM.